New approaches to cystic fibrosis

Better understanding of the pathogenesis of cystic fibrosis-how the chain of causality moves from gene to tissue to organ-has led to therapeutic approaches that attack virtually every stage of this process. Within the coming decade, it is realistic to anticipate that the disease will be controlled via somatic gene therapy in children and young adults. Farther down the road, but already technically feasible, lies the possibility of real prevention through genetic intervention in utero or soon after birth.